Results 41 to 50 of about 31,702 (234)
Bleeding, Thrombosis and Vascular BiologyDear Editor, I wish to report that the first gene therapy procedure in Italy for the treatment of severe or moderately severe hemophilia B has been successfully administered at the Policlinico of Milan....
Pier Mannuccio Mannucci
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Report on the diagnosis and treatment of hemophilia in China (Version 2024)
LabMed DiscoveryHemophilia is a hemorrhagic disease caused by a lack of clotting factors due to genetic mutations, and is a representative of rare diseases. The prevalence rate of hemophilia is about 2.73/100,000–3.09/100,000 in China, and the absolute number of ...
Feng Xue +11 more
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Emicizumab Associated Rhabdomyolysis in Hemophilia A
Clinical Hematology International, 2020 Emicizumab is increasingly the front-line treatment for patients with Hemophilia A with or without inhibitors. Rhabdomyolysis is a syndrome of muscle necrosis and release of intracellular muscle constituents into the circulation.
Joseph A. Wilson +5 more
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Porcine model of hemophilia A. [PDF]
PLoS ONE, 2012 Hemophilia A is a common X chromosome-linked genetic bleeding disorder caused by abnormalities in the coagulation factor VIII gene (F8). Hemophilia A patients suffer from a bleeding diathesis, such as life-threatening bleeding in the brain and harmful ...
Yuji Kashiwakura +15 more
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Journal of Employment Counseling, EarlyView.ABSTRACT This study explores how social capital and chronic illness self‐management influence career development competency among young people with chronic disabling health conditions (YPCDHC) in Hong Kong. Using data from 485 participants aged 15–29, path analysis showed that school/workplace social capital had the strongest positive effects on career
Steven Sek‐yum Ngai +10 more
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Neuroprotection, EarlyView.Abstract Prime editing, a novel clustered regularly interspaced short palindromic repeats (CRISPR)‐based technology, fuses a reverse transcriptase (RT) to an engineered CRISPR‐associated protein 9 (Cas9) and uses a prime editing guide RNA (pegRNA)‐encoded template.
Tianshan Ji +4 more
wiley +1 more source
MOLECULAR MECHANISMS OF INHIBITOR DEVELOPMENT IN HEMOPHILIA
Mediterranean Journal of Hematology and Infectious Diseases, 2020 The development of neutralizing antibodies in hemophilia is a serious complication of factor replacement therapy. These antibodies, also known as “inhibitors”, significantly increase morbidity within the hemophilia population and lower the quality of ...
Davide Matino +2 more
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Hemophilia A: An Ideal Disease for Prenatal Therapy
Prenatal Diagnosis, EarlyView.ABSTRACT Hemophilia A (HA) is the most common inherited coagulation defect. Current state‐of‐the‐art treatment consists of frequent administration of prophylactic infusions of coagulation factor VIII (FVIII) protein or bispecific antibodies that replace the cofactor function of FVIIIa to maintain hemostasis. However, these treatments are far from ideal,
Christopher D. Porada +2 more
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Journal of Applied Hematology, 2021 Spontaneous spinal extradural hematomas are rarely attributed to hemophilia. When it occurs, magnetic resonance imaging (MRI) best establishes the diagnosis that should prompt immediate medical or surgical management.
Ashish Acharya +3 more
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Cross Sectional Study of Prenatal Diagnosis Uptake Among Individuals With Genetic Conditions
Prenatal Diagnosis, EarlyView.ABSTRACT Objective Prenatal diagnostic genetic testing allows for early identification of significant fetal conditions and enables informed decision‐making regarding management options. The aim of this study was to assess prenatal testing practice among individuals with genetic conditions.
Ebunoluwa Ojo +4 more
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